Merck Announces Orphan Drug Designation for Investigational Therapy Tepotinib in Patients with NSCLC Harboring MET Gene Alterations

- Japanese Ministry of Health, Labour and Welfare grants orphan
drug designation for diseases that affect fewer than 50,000 patients
in Japan, and for which significant unmet medical need exists

- MET exon 14 (METex14) skipping alterations and MET
amplifications are present in 3-5% of non-small cell lung cancer
patients and correlate with poor prognosis

- In March 2018, tepotinib received SAKIGAKE 'fast-track'
regulatory designation in Japan and in September 2019 received
Breakthrough Therapy Designation in the US

Darmstadt, Germany (ots/PRNewswire) - Merck, a leading science and
technology company, today announced that the Japanese Ministry of
Health, Labour and Welfare (MHLW) has granted orphan drug designation
(ODD) for its investigational therapy tepotinib* for patients with
non-small cell lung cancer (NSCLC) harboring MET gene alterations.

"Advanced NSCLC harboring MET gene alterations is associated with
aggressive tumor behavior and poor clinical prognosis," said Luciano
Rossetti, Global Head of Research & Development for the Biopharma
business of Merck. "This orphan drug designation helps to advance
this program within Japan and, coupled with the SAKIGAKE 'fast-track'
designation received last year, provides important mechanisms, such
as priority review, to quickly deliver this medicine to Japanese
patients with this difficult-to-treat disease."

The Japan MHLW ODD program is designed to promote research and
development of orphan drugs for diseases that affect fewer than
50,000 patients in Japan, and for which significant unmet medical
need exists. An investigational drug can qualify for ODD if there is
no approved alternative treatment option or if there is an
expectation of high efficacy or safety compared to existing treatment
options. Drugs receiving ODD qualify for several benefits intended to
support development, such as guidance and subsidies for research and
development activities from the MHLW, preferential tax treatment,
priority consultation for clinical development, and priority review
of applications.

Discovered in-house at Merck, tepotinib is an investigational oral
MET inhibitor that is designed to inhibit the oncogenic MET receptor
signaling caused by MET (gene) alterations, including both MET exon
14 skipping alterations and MET amplifications, or MET protein
overexpression. It has been designed to have a highly selective
mechanism of action,1 with the potential to improve outcomes in
aggressive tumors that have a poor prognosis and harbor these
specific alterations.

Alterations of the MET signaling pathway are found in various
cancer types, including 3-5% of NSCLC cases, and correlate with
aggressive tumor behavior and poor clinical prognosis.2-4 Lung cancer
is the most common type of cancer worldwide, with two million cases
diagnosed annually.5

Tepotinib is being investigated in the ongoing VISION study
(NCT02864992), which showed preliminary efficacy in patients
harboring METex14 skipping alterations detected by liquid biopsy
(LBx) or tissue biopsy (TBx) across different lines of treatment.

Results from the interim analysis of the VISION study were
presented in an oral presentation at the 2019 American Society of
Clinical Oncology (ASCO) Annual Meeting6 and the 2019 Japan Society
of Medical Oncology (JSMO) Annual Meeting.7 The use of both LBx and
TBx to identify patients for the VISION study is intended to support
improved patient selection and is consistent with the company's focus
on patient-centric drug development.

Tepotinib is also being investigated in the INSIGHT 2 study
(NCT03940703) in combination with the tyrosine kinase inhibitor (TKI)
osimertinib in epidermal growth factor receptor (EGFR) mutated, MET
amplified, locally advanced or metastatic NSCLC having acquired
resistance to prior EGFR TKI.

In March 2018, the Japan MHLW granted SAKIGAKE 'fast-track'
designation for tepotinib in advanced (stage IIIB/IV) NSCLC harboring
METex14 skipping alterations and, in September 2019, the US Food and
Drug Administration (FDA) granted Breakthrough Therapy Designation
(BTD) for tepotinib in patients with metastatic NSCLC harboring
METex14 skipping alterations who progressed following platinum-based
cancer therapy.

*Tepotinib is currently under clinical investigation and not
approved for any use anywhere in the world.

About Non-Small Cell Lung Cancer

With two million cases diagnosed annually, lung cancer (including
trachea, bronchus and lung) is the most common type of cancer
worldwide, and the leading cause of cancer-related death, with 1.7
million mortality cases worldwide.5 Alterations of the MET signaling
pathway, including MET exon 14 skipping alterations and MET
amplifications, occur in 3-5% of NSCLC cases.2-4

About Tepotinib

Tepotinib, discovered in-house at Merck, is an investigational
oral MET inhibitor that is designed to inhibit the oncogenic MET
receptor signaling caused by MET (gene) alterations, including both
MET exon 14 skipping alterations and MET amplifications, or MET
protein overexpression. It has been designed to have a highly
selective mechanism of action,1 with the potential to improve
outcomes in aggressive tumors that have a poor prognosis and harbor
these specific alterations.

Tepotinib is currently being investigated in NSCLC and Merck, is
actively assessing the potential of investigating tepotinib in
combination with novel therapies and in other tumor indications.

References

1. Bladt, F, et al. Clin Cancer Res 2013;19:2941-2951
2. Reungwetwattana T, et al. Lung Cancer 2017;103:27-37.
3. Mo HN, et al. Chronic Dis Transl Med 2017; 3(3):148-153.
4. Lutterbach B, et al. Cancer Res 2007;67:2081-8.
5. Bray F, et al. CA Cancer J Clin. Global cancer statistics 2018:
GLOBOCAN estimates of incidence and mortality worldwide for 36
cancers in 185 countries. 2018;68(6):394-424.
https://doi.org/10.3322/caac.21492 PMID:30207593
6. Paik P, et al. J Clin Oncol 2019;37: (suppl; abstr 9005).
7. Sakai H, et al. Ann Oncol 2019;30(Suppl_6):Abstract MO2-15-5.

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Contact:
annemarie.eckhardt@merckgroup.com
Phone: +49 6151 72 26560

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